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1.
Impaired cholesterol efflux in retinal pigment epithelium of individuals with juvenile macular degeneration.
Am J Hum Genet
; 108(5): 903-918, 2021 05 06.
Article
in English
| MEDLINE | ID: mdl-33909993
2.
A novel pathogenic CRB1 variant presenting as Leber Congenital Amaurosis 8 and evaluation of gene editing feasibility.
Doc Ophthalmol
; 147(3): 217-224, 2023 12.
Article
in English
| MEDLINE | ID: mdl-37804373
3.
CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa.
Mol Ther
; 30(4): 1407-1420, 2022 04 06.
Article
in English
| MEDLINE | ID: mdl-35150888
4.
Analysis of CRB1 Pathogenic Variants Correctable with CRISPR Base and Prime Editing.
Adv Exp Med Biol
; 1415: 103-107, 2023.
Article
in English
| MEDLINE | ID: mdl-37440021
5.
Generation of CRB1 RP Patient-Derived iPSCs and a CRISPR/Cas9-Mediated Homology-Directed Repair Strategy for the CRB1 c.2480G>T Mutation.
Adv Exp Med Biol
; 1415: 571-576, 2023.
Article
in English
| MEDLINE | ID: mdl-37440088
6.
Prime Editing Strategy to Install the PRPH2 c.828+1G>A Mutation.
Adv Exp Med Biol
; 1415: 97-102, 2023.
Article
in English
| MEDLINE | ID: mdl-37440020
7.
Generation of an Avian Myeloblastosis Virus (AMV) Reverse Transcriptase Prime Editor.
Adv Exp Med Biol
; 1415: 109-114, 2023.
Article
in English
| MEDLINE | ID: mdl-37440022
8.
Loss of CRB2 in Müller glial cells modifies a CRB1-associated retinitis pigmentosa phenotype into a Leber congenital amaurosis phenotype.
Hum Mol Genet
; 28(1): 105-123, 2019 01 01.
Article
in English
| MEDLINE | ID: mdl-30239717
9.
The Role of Small Molecules and Their Effect on the Molecular Mechanisms of Early Retinal Organoid Development.
Int J Mol Sci
; 22(13)2021 06 30.
Article
in English
| MEDLINE | ID: mdl-34209272
10.
Defining Phenotype, Tropism, and Retinal Gene Therapy Using Adeno-Associated Viral Vectors (AAVs) in New-Born Brown Norway Rats with a Spontaneous Mutation in Crb1.
Int J Mol Sci
; 22(7)2021 Mar 30.
Article
in English
| MEDLINE | ID: mdl-33808129
11.
Mouse Models of Achromatopsia in Addressing Temporal "Point of No Return" in Gene-Therapy.
Int J Mol Sci
; 22(15)2021 Jul 28.
Article
in English
| MEDLINE | ID: mdl-34360834
12.
CRB2 in immature photoreceptors determines the superior-inferior symmetry of the developing retina to maintain retinal structure and function.
Hum Mol Genet
; 27(18): 3137-3153, 2018 09 15.
Article
in English
| MEDLINE | ID: mdl-29893966
13.
Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models.
Hum Mol Genet
; 24(11): 3104-18, 2015 Jun 01.
Article
in English
| MEDLINE | ID: mdl-25701872
14.
Targeted ablation of CRB1 and CRB2 in retinal progenitor cells mimics Leber congenital amaurosis.
PLoS Genet
; 9(12): e1003976, 2013.
Article
in English
| MEDLINE | ID: mdl-24339791
15.
Targeting miR-181a/b in retinitis pigmentosa: implications for disease progression and therapy.
Cell Biosci
; 14(1): 64, 2024 May 21.
Article
in English
| MEDLINE | ID: mdl-38773556
16.
Generation of Human iPSC-Derived Retinal Organoids for Assessment of AAV-Mediated Gene Delivery.
Methods Mol Biol
; 2560: 287-302, 2023.
Article
in English
| MEDLINE | ID: mdl-36481905
17.
Culture of Human Retinal Explants for Ex Vivo Assessment of AAV Gene Delivery.
Methods Mol Biol
; 2560: 303-311, 2023.
Article
in English
| MEDLINE | ID: mdl-36481906
18.
Clinical and Therapeutic Evaluation of the Ten Most Prevalent CRB1 Mutations.
Biomedicines
; 11(2)2023 Jan 27.
Article
in English
| MEDLINE | ID: mdl-36830922
19.
Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1KO and CRB1KOCRB2+/- retinal organoids.
Mol Ther Methods Clin Dev
; 31: 101128, 2023 Dec 14.
Article
in English
| MEDLINE | ID: mdl-37886604
20.
Prime Editing for the Installation and Correction of Mutations Causing Inherited Retinal Disease: A Brief Methodology.
Methods Mol Biol
; 2560: 313-331, 2023.
Article
in English
| MEDLINE | ID: mdl-36481907